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FDA approves ORFADIN® (nitisinone) Oral Suspension

WALTHAM, Mass., April 26, 2016 /CNW/ -- Swedish Orphan Biovitrum AB (Sobi), an international specialty healthcare company dedicated to rare diseases, today announced that the Food and Drug Administration (FDA) has approved ORFADIN® Oral Suspension for the treatment of Hereditary Tyrosinemia Type 1 (HT-1). ORFADIN is indicated for use in combination with dietary restriction of tyrosine and phenylalanine.

This new formulation is an alternative to the capsules and provides an additional option for infants and small children. ORFADIN Oral Suspension comes in a 4 mg/mL dose.

"We continue to support the HT-1 community with the introduction of the oral suspension. This new liquid formulation provides an additional option for parents with small children taking ORFADIN, or for anyone who has difficulty swallowing capsules," said Len Walt, Vice President & Head of Medical Affairs, North America.

HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver and kidney complications and can be fatal if untreated. Before pharmacological treatment was available, less than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.1 Treatment with ORFADIN combined with dietary restriction of tyrosine and phenylalanine plus more widespread newborn screening leading to early diagnosis has greatly improved outcomes for HT-1 patients.2

ORFADIN Oral Suspension will be available in August 2016.

About ORFADIN®

People with Hereditary Tyrosinemia type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver, renal and neurological complications. ORFADIN® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic by-products in the body. In the most common form of the disease, symptoms arise within the first six months of the child's life. Patients must maintain a special diet in combination with ORFADIN treatment as tyrosine is not adequately broken down. Approximately 1,000 persons are identified as living with HT-1 today. ORFADIN is a proprietary product and is developed by and marketed globally by Sobi. For more information about ORFADIN, visit www.orfadin.com.

Indication and Usage

ORFADIN is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).

Important Safety Information

Inadequate restriction of tyrosine and phenylalanine intake can result in elevations in plasma tyrosine.

Do not adjust ORFADIN dosage in order to lower the plasma tyrosine concentration.

Leucopenia and thrombocytopenia have been observed with treatment with ORFADIN. Monitor platelet and white blood cell counts regularly during ORFADIN therapy.

Use caution when administering ORFADIN with drugs that are metabolized by CYP2C9 because of a potential for increased systemic exposure of these drugs.

ORFADIN should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Based on animal data, ORFADIN may cause fetal harm.

Exercise caution when administering to a nursing woman. Because of the potential for serious adverse reactions in nursing infants from ORFADIN, a decision should be made to discontinue nursing or to discontinue the drug taking into account the importance of the drug to the mother.

Sobi™ is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. We also market a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2015, Sobi had total revenues of SEK 3.2 billion (USD 385 M) and approximately 700 employees. The share (STO:SOBI) is listed on NASDAQ Stockholm. More information is available on www.sobi.com.